Effect of imetelstat on genotype of hematopoietic colonies (CFU-GM) assayed from MF CD34+ cells
| . | Cytokines + vehicle . | Cytokines + MM (7.5 μM) . | Cytokines + Ime (7.5 μM) . | |||
|---|---|---|---|---|---|---|
| % JAK2V617F . | % Homozygous JAK2V617F . | % JAK2V617F . | % Homozygous JAK2V617F . | % JAK2V617F . | % Homozygous JAK2V617F . | |
| Pt 5 | 38 (6/16)* | 31 (5/16) | 56 (15/27) | 52 (14/27) | 9 (2/23) | 0 (0/23) |
| Pt 6 | 100 (27/27) | 89 (24/27) | 100 (22/22) | 82 (18/22) | 100 (30/30) | 97 (29/30) |
| Pt 7 | 64 (9/14) | 36 (5/14) | 72 (13/18) | 22 (4/18) | 60 (12/20) | 20 (4/20) |
| Pt 8 | 100 (20/20) | 80 (16/20) | 100 (21/21) | 90 (19/21) | 100 (11/11) | 73 (8/11) |
| Pt 12 | 63 (19/30) | 37 (11/30) | 80 (20/25) | 56 (14/25) | 82 (25/31) | 39 (12/31) |
| Pt 13 | 96 (25/26) | 81 (21/26) | 86 (19/22) | 68 (15/22) | 70 (14/20) | 50 (10/20) |
| . | Cytokines + vehicle . | Cytokines + MM (7.5 μM) . | Cytokines + Ime (7.5 μM) . | |||
|---|---|---|---|---|---|---|
| % JAK2V617F . | % Homozygous JAK2V617F . | % JAK2V617F . | % Homozygous JAK2V617F . | % JAK2V617F . | % Homozygous JAK2V617F . | |
| Pt 5 | 38 (6/16)* | 31 (5/16) | 56 (15/27) | 52 (14/27) | 9 (2/23) | 0 (0/23) |
| Pt 6 | 100 (27/27) | 89 (24/27) | 100 (22/22) | 82 (18/22) | 100 (30/30) | 97 (29/30) |
| Pt 7 | 64 (9/14) | 36 (5/14) | 72 (13/18) | 22 (4/18) | 60 (12/20) | 20 (4/20) |
| Pt 8 | 100 (20/20) | 80 (16/20) | 100 (21/21) | 90 (19/21) | 100 (11/11) | 73 (8/11) |
| Pt 12 | 63 (19/30) | 37 (11/30) | 80 (20/25) | 56 (14/25) | 82 (25/31) | 39 (12/31) |
| Pt 13 | 96 (25/26) | 81 (21/26) | 86 (19/22) | 68 (15/22) | 70 (14/20) | 50 (10/20) |
Individual colonies (CFU-GM) from 6 JAK2V617F+ MF patients (JAK2V617F allele burden: 25%-90%) treated with cytokines alone or cytokines plus imetelstat or MM (7.5 μM) were plucked and genotyped for the JAK2V617F. In 3 of 4 patients (Pts 5, 7, 12, 13) having a reservoir of wild-type JAK2, imetelstat treatment reduced the percent of JAK2V617F+ colonies and percent of homozygous JAK2V617F+ colonies generated.
The numbers in parentheses denote the actual number of total JAK2V617F+ or homozygous JAK2V617F+ CFU-GM/the total numbers of CFU-GM plucked and genotyped.